Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
View Patent ↗The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. Specifically, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver short interfering nucleic acid (siNA). The compositions described herein are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).
1. A lipid nanoparticle (LNP) composition comprising one or more short interfering nucleic acid (siNA) molecules, 3-Dimethylamino-2-(Cholest-5-en-3β-oxybutan-4-oxy)-1-(cis,cis-9,12-octadecadienoxy) propane (CLinDMA), distearoylphosphatidylcholine (DSPC), Cholesterol, and 1-[8′-(1,2-Dimyristoyl-3-propanoxy)-carboxamido-3′,6′-dioxaoctanyl]carbamoyl-ω-methyl-poly(ethylene glycol) (PEG-DMG).
2. The LNP composition of claim 1 , further comprising Linoleyl alcohol.
3. The LNP composition of claim 2 , wherein said CLinDMA, DSPC, Cholesterol, PEG-DMG, and Linoleyl alcohol have a molar ratio of 43/36/10/4/7.
4. The LNP composition of claim 1 , wherein said siNA molecule comprises one or more ribonucleotides.
5. The LNP composition of claim 1 , wherein said siNA molecule comprises a sense strand and an antisense strand.
6. The LNP composition of claim 5 , wherein one or more purine nucleotides present in said sense strand are 2′-deoxy purine nucleotides.
7. The LNP composition of claim 5 , wherein one or more purine nucleotides present in said antisense strand are 2′-O-methyl purine nucleotides.
8. The LNP composition of claim 5 , wherein one or more pyrimidine nucleotides present in said sense strand are 2′-deoxy-2′-fluoro nucleotides.
9. The LNP composition of claim 5 , wherein one or more pyrimidine nucleotides present in said antisense strand are 2′-deoxy-2′-fluoro nucleotides.
10. A composition comprising the LNP composition of claim 1 in a pharmaceutically acceptable carrier or diluent.